去纤苷获批适应症有什么？

Defibrotide (defiteli) was first developed by Gentium Pharmaceuticals of Italy. It was approved for marketing in the European Union in October 2013. It was approved for marketing in the United States by the U.S. Food and Drug Administration (FDA) on March 30, 2016. It is a U.S. F The first drug approved by the DA to treat severe hepatic veno-occlusive disease is used for adult or pediatric patients with hepatic venous obstruction (VOD) and renal or lung function abnormalities after receiving blood or bone marrow hematopoietic stem cell transplantation (HSCT). The drug also received "orphan drug" designation. 

Defibrotide (defiteli) is an adenosine receptor agonist with multiple effects. The adenosine A1/A2 receptors of endothelial cells are involved in the regulation of endothelial cells and the response of endothelial cells to injury. Defibrotide (defiteli) can act on these receptors to produce a variety of downstream effects. Defibrotide (defiteli) can also reduce the expression of cell adhesion molecules on the surface of endothelial cells, thereby reducing the adhesion of leukocytes to vascular endothelial cells and reducing the inflammatory damage of endothelial cells.

The recommended dose of defibrotide (defiteli) is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. Treatment lasts for a minimum of 21 days. If signs and symptoms of VOD do not resolve after 21 days, continue treatment until resolution. Hypersensitivity reactions have occurred in less than 2% of patients treated with defibrotide (defiteli). These reactions include rash, urticaria, and angioedema. If a severe hypersensitivity reaction occurs, discontinue defiteli, treat with standard medical care, and monitor until symptoms resolve.

In one study, 19 patients with sHOVD were included. The dose of defibrotide (defiteli, defiteli) is 5~60 mg/kg per day, intravenous infusion, and the median usage time is 15 days (2-61 days). The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%, and the patients could tolerate defibrination (defiteli) well. Since then, a number of clinical trials have confirmed the effectiveness of defibrotide (defiteli) in the treatment of hepatic veno-occlusive disease.